PGI61 REGULATORY AND HTA ENGAGEMENT IN NEURODEGENERATIVE DISEASE RESEARCH: A DECISION TOOL

Timely and effective engagement with regulatory and HTA agencies on evidence requirements is considered best practice in ensuring that their requirements are included in the evidence development strategy to support new treatments. This is particularly relevant in the area of neurodegenerative disease, for which despite high levels of research investment, there remains a lack of effective treatments. Neuronet aims to address this challenge for a portfolio of Innovative Medicines Initiative (IMI) neurodegenerative disease projects. Through the development of guidance, Neuronet will enable meaningful engagement between projects and regulatory and HTA agencies in order to maximise the usability of project outputs across the EU regulatory and HTA framework. We developed a decision tool for use by European research projects in the neurodegenerative disease area that identifies what form of engagement with regulatory and HTA agencies would be best suited, depending on the nature of the question at hand, product development stage, and intended use of the treatment. Specifically, the tool provides advice and considerations on informal routes for engagement as well as the formal procedures that are available at key stages along the research and development pathway. Whilst the tool has been based around the needs of and categories of research being undertaken in the current IMI neurodegenerative disease portfolio, it is intended that the guidance will be applicable beyond the scope of this work. The decision tool provides a practical framework to guide engagement between researchers, regulatory and HTA agencies in the neurodegenerative disease area. By engaging with these key stakeholders at relevant stages of evidence development, researchers will be better placed for developing outputs and evidence which can be translated into practice, thereby improving access to new treatments for people with neurodegenerative diseases in the future.